Capsida has received FDA IND clearance to initiate clinical trials for our IV-administered investigational gene therapies for Parkinson’s Disease associated with GBA mutations (PD-GBA) and STXBP1 Developmental and Epileptic Encephalopathy (STXBP1-DEE). For more information about the trials click here (NCT07011771 and NCT06983158).




UNLOCKING THE POTENTIAL OF GENE THERAPY FOR ALL
About
Capsida is a clinical stage, fully integrated next-generation genetic medicines company advancing potentially disease modifying and curative treatments for rare and more common diseases
“We are focused on bringing our innovative therapies to the people who so desperately need them. Our investigational therapies for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE) and Parkinson’s disease caused by GBA mutations (PD-GBA) recently received FDA IND clearance and we are now initiating clinical trials.”
Peter Anastasiou, CEO
Capsida Biotherapeutics is developing next generation, targeted, IV-administered genetic medicines for rare and more common diseases across all ages. The Investigational New Drug (IND) applications for our potential first and best-in-class investigational therapy for STXBP1-DEE, and for our potential best-in-class investigational therapy for PD-GBA, have received FDA clearance and we are now initiating clinical trials. We are also working on a potential best-in-class investigational therapy for Friedreich’s ataxia (FA), which is currently in IND-enabling studies.
Pipeline
Our Pipeline is Making the Impossible Possible
Capsida’s intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels in desired tissues and cells, while limiting tropism to non-target organs, such as the liver and dorsal root ganglion (DRG).
Our investigational therapies for STXBP1-DEE and PD-GBA have received IND clearance from FDA, and we are initiating clinical trials. These are the first engineered, IV-delivered gene therapies that cross the blood-brain-barrier and detarget the liver and dorsal root ganglia (DRG) to enter clinical development.
Our third program is in IND-enabling studies for FA and is aiming to target CNS, cardiac, and sensory manifestations with a single IV infusion.
In our partnered programs with AbbVie, CRISPR Therapeutics, and Lilly’s Gene Therapy Division, we have the potential to co-develop and co-commercialize up to three CNS programs. Partnered programs target CNS and ophthalmology disease indications.
Science & Innovation
THE CAPSIDA APPROACH
Capsid Engineering, Cargo Optimization, Translational Biology, Process Development and Good Manufacturing Practices (GMP) Manufacturing, and Clinical Development – All Under One Roof to Develop Better Therapies Faster
Capsida Biotherapeutics is the only clinical-stage fully integrated next-generation genetic medicines company that has created all the capabilities needed to deliver on the promise of genetic medicine.

Capsid Engineering
At Capsida, the scale at which we can engineer and screen capsids is unrivaled. Using our automated screening method, our scientists assess large, diverse engineered capsid libraries in non-human primates to quickly identify optimal capsids for disease targets and their corresponding human receptors.

Discovery and Preclinical
To quickly advance our mission of providing meaningful therapeutic opportunities for patients with unmet medical need, Capsida optimizes cargo and evolves preclinical disease proof-of-concept in parallel with capsid engineering. Our engineered capsids can be optimized to deliver a broad range of cargo modalities.

GMP Manufacturing Operations
At Capsida, we are singularly focused on rapidly getting our high-quality, life-changing therapies to patients who are suffering from debilitating diseases. We have built state-of-the-art cGMP manufacturing capabilities to perform everything in-house. We streamline the entire process, from selecting our therapies for manufacturability through scalable process development. Ultimately, this helps us get therapeutics to patients sooner.

Clinical Development
Capsida’s approach unlocks the potential to treat both rare and common diseases across all ages. First and foremost, we approach patients holistically, working closely with advocacy groups, patients, and caregivers to ensure we are meeting their needs.
Team
Capsida’s founding technology originated in the lab of Caltech’s Viviana Gradinaru, Ph.D., Professor of Neurosciences and Biological Engineering at the Tianqiao and Chrissy Chen Institute of Neuroscience. Founded in 2019, Capsida is financed by Versant Ventures and Westlake Village BioPartners and has entered into strategic collaborations with AbbVie, CRISPR Therapeutics, Lilly’s Gene Therapy Division, and Kate Therapeutics (acquired by Novartis).
Our leaders have decades of experience in the biopharmaceutical industry and expertise in AAV biology and biologics manufacturing.
Partnering
“Our partnerships with leading global biopharmaceutical companies are key to Capsida achieving its vision of being able to reach more potential patients in need with our enabling genetic medicines.”
Bethany Mancilla, Chief Business Officer
Our goal is to apply the strength of our targeted genetic medicine platform to develop a new generation of transformative therapies. Since our inception, we have entered into strategic collaborations with AbbVie, CRISPR Therapeutics, Lilly’s Gene Therapy Division, and Kate Therapeutics (acquired by Novartis).
Careers
BE PART OF OUR DNA
Capsida is a vibrant company based in the heart of the Greater Los Angeles biotech hub. We’re looking for passionate, dedicated colleagues to join us!
News & Events
CAPSIDA NEWS & EVENTS
June 11, 2025
Capsida Receives FDA IND Clearance for Its IV-Administered Gene Therapy for Parkinson’s Disease Associated with GBA Mutations
Capsida is initiating the Phase 1/2 study for CAP-003, with the first patient expected to be dosed in the third quarter of this year THOUSAND OAKS, Calif.,…
May 29, 2025
Capsida Receives FDA Fast Track Designation for Its Potential First-in-Class IV-Administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
Capsida previously received FDA Orphan Drug Designation and IND clearance to initiate the SYNRGY Phase 1/2a clinical trial for CAP-002 THOUSAND OAKS, Calif.,…